[Congressional Record (Bound Edition), Volume 158 (2012), Part 1]
[House]
[Pages 1138-1139]
[From the U.S. Government Publishing Office, www.gpo.gov]




       INCREASE FUNDING FOR RARE DISEASE RESEARCH AND DEVELOPMENT

  The SPEAKER pro tempore. The Chair recognizes the gentleman from New 
York (Mr. Towns) for 5 minutes.
  Mr. TOWNS. Mr. Speaker, recently I met Jill Wood from my district in 
Brooklyn. I was very moved by this meeting as she described the 
struggles of her son, who is diagnosed with a rare genetic disease 
known as Sanfilippo syndrome.
  Before we met, I was not familiar with this particular disease, but 
she touched my heart to hear about her child's courage. Every day he 
has to overcome physical disabilities that make it almost impossible 
for him to complete a very simple task that we complete with ease. I 
was inspired by the strength of their family and the bond that they 
share.
  I have long been a strong advocate for rare disease research and 
development. In fact, this is why I am working with my colleague from 
Florida, Congressman Stearns, on H.R. 3737, the ULTRA Act. This bill 
would codify the flexibility the FDA needs to encourage development of 
treatments for rare diseases like Sanfilippo syndrome.
  It is our duty, as Members of the United States Congress, to come 
together and support measures that aid

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the rare disease community. Imagine being afflicted with a disease your 
physician has never heard of and has no idea as to how to treat it. Can 
you imagine the devastation this would cause to your family?
  We must provide the National Institutes of Health with additional 
funding to support the important research for orphan and rare diseases. 
We must also give flexibility and support to the FDA to help the agency 
bring potential cures and treatments to the market much sooner.
  How long must we wait and continue to suffer until lifesaving 
treatments are available?
  Nearly 30 million Americans are affected by 7,000 rare diseases. We 
must do everything in our power to support education, advocacy, 
research, and patient assistance to bring this number down. Imagine the 
families out there watching their loved ones suffer because we have not 
yet provided enough support for this cause.
  We are a great Nation of innovation, but that innovation and drive 
only goes so far without the proper support coming from the government. 
Our constituents need to know that we hear their needs and that, as 
their elected officials, we are determined to make available the 
resources that will support them.
  The next time I speak to a family affected by rare diseases, I want 
to be able to look them in the eyes and tell them that we have helped, 
that we made available the means necessary to support lifesaving 
research and development, we care and we will do everything in our 
power to ensure that everyone has the chance to live full, healthy, and 
prosperous lives.
  Thank you, Jill, for bringing this to my attention.
  I urge my colleagues to support the ULTRA Act and increase funding 
for rare disease research and development. It is so important that we 
do everything possible to be able to bring the numbers down.

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